The U.S. Food and Drug Administration granted various designations for Sanofi’s avalglucosidase alfa for Pompe disease, Sanofi’s rilzabrutinib for immune thrombocytopenia, and ViiV Healthcare’s cabotegravir for HIV prevention.

San Diego-based Retrophin is buying Orphan Technologies for $90 million in cash up front and up to $427 million in milestone payments.

Shire’s experimental treatment SHP609 to address cognitive impairment in pediatric patients diagnosed with the rare disease known as Hunter syndrome failed a key trial.

Barely a week after announcing it was moving into a new corporate headquarters, Sangamo Therapeutics announced that the company has dosed the first patient in its Phase I/II gene therapy trial.